Article
Genome editing using CRISPR-Cas9 to create the HPFH genotype in HSPCs: An approach for treating sickle cell disease and β-thalassemia.
Proceedings of the National Academy of Sciences of the United States of America - 20 Sept 2016
Ye Lin, Wang Jiaming, Tan Yuting, Beyer Ashley I, Xie Fei, Muench Marcus O, Kan Yuet Wai
Abstract excerpt
Hereditary persistence of fetal hemoglobin (HPFH) is a condition in some individuals who have a high level of fetal hemoglobin throughout life. Individuals with compound heterozygous β-thalassemia or sickle cell disease (SCD) and HPFH have milder clinical manifestations. Using RNA-guided clustered regularly interspaced short palindromic repeats-associated Cas9 (CRISPR-Cas9) genome-editing technology, we deleted,...
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